“We have devoted our scientific discoveries and our tireless pursuits for the patients and families afflicted with SMA. We have every reason to be excited about advancing towards human trials and the potential to make a clinical meaningful impact on this disease. Our team will not rest until we have made a difference.” – Brian K. Kaspar

“Spinal Muscular Atrophy has forever changed our lives. It has taught our family to cherish every experience we share together. We created The Sophia’s Cure Foundation to raise awareness of SMA and to find a cure for this deadly disease. If we could help just one family save their child, it would all be worth it. I have learned to live by one motto, Reality may control the time…….but we will always control the moments.” – The Gaynor Family

Spinal Muscular Atrophy (SMA) is an autosomal recessive motor neuron disease. The gene Survival Motor Neuron (SMN1) is mutated in patients with SMA. This mutation reduces the level of functional SMN protein in the patient. In the absence of SMN protein, motor neurons begin dying. The degeneration of motor neurons in the spinal cord and brainstem leads to muscle weakness, often observed as weakness in controlling head and neck movements. Muscles controlling...

“SMA has had a tremendous impact on our family. It changes the way you look at life. The small things become treasured memories that we took for granted before Avery was diagnosed. We decided to start Avery’s Race to CURE SMA in July of 2010 to support Dr. Kaspar’s gene therapy program because we feel he has the most promising research.” – The Pitzen Family

Cure SMA funds and directs the leading SMA research programs to develop a treatment and cure for the disease. The successful results and progress that the organization has delivered, from basic research to drug discovery to clinical trials, provide real hope for families and patients impacted by the disease. The charity has invested over $55 million in research and has been involved in funding half of all the ongoing novel drug programs for SMA. Each year,...

At AveXis, we are always looking for talented and dedicated people to join our team. Based in Dallas, Texas, AveXis is a clinic-stage gene therapy platform company establishing unique industry and academic alliances to create innovative treatments for people with unmet medical needs.

Dallas – February 25, 2014 – AveXis, Inc., a biotechnology company focused on using gene therapy to transform the lives of patients with severe genetic and orphan diseases like Spinal Muscular Atrophy (SMA), today announced the formation of its EU-based wholly-owned subsidiary…

AveXis is dedicated to supporting families who are affected by Spinal Muscular Atrophy. We are proud sponsors of organizations like Cure SMA, The Sophia’s Cure Foundation, and Avery’s Race who are trying to spread awareness and support to communities so that we can find a treatment for SMA.